The project addresses a long-standing gap in the field: the lack of a clear human reference standard for muscle regeneration. A new research collaboration between muscular dystrophy organizations aims ...

Obstructive sleep apnea prevalence increased in children with achondroplasia beginning at school age (6-12 years) and remained elevated into adolescence. Recurrent otitis media may accompany ...

Between 2001 to 2011, annual age-adjusted incidence increased from 0.55/100,000 to 0.78/100,000, demonstrating an increasing trend with age. Incidence peaks in individuals over 70 years of age ...

Life expectancy in DMD has improved over time. Previous estimates pointed to a life expectancy of 25 years, with more recent data reporting a life expectancy of 31.7 years (95% CI, 27.4-36.0). 3,4 A ...

Patients with MG who took C5 inhibitors saw greater reductions in concomitant OCS and NSIST use than did those who took efgartigimod. Rare Disease Advisor, a trusted source of medical news and feature ...

At present, no validated tools or scoring systems have been developed to predict prognosis or clinical course of warm autoimmune hemolytic anemia (AIHA). Several potential prognostic risk factors that ...

Chronic inflammatory demyelinating polyneuropathy (CIDP) is a rare acquired autoimmune neurological disorder in which both T-cell-mediated and humoral immune mechanisms target healthy myelinated ...

Individuals with advanced liver disease may also experience significant weight loss due to nausea and poor appetite. Candidates for liver transplantation may need to gain weight to optimize surgical ...

US and European regulators could approve up to 17 gene therapies this year, with a top official at the US Food and Drug Administration (FDA) predicting that 2024 will be a “breakout year” in ...

(L-R) Rare Disease Advisor senior correspondent Larry Luxner, patient columnists Alithea Athans, Tara Keith and Tom Bartlett, and director of advocacy relations Vera Luxner (Photo by Riya Ajmera) ...

Inebilizumab-cdon (Uplizna ®) has received US Food and Drug Administration (FDA) approval for the treatment of adults with generalized myasthenia gravis (MG) with antibodies against the acetylcholine ...

A new phase 2 clinical trial to test the safety, efficacy, and tolerability of TAK-411 for the treatment of chronic inflammatory demyelinating polyneuropathy (CIDP) is now open. The open-label, ...